ALS Compendium

Title: Expansive gene transfer in the rat CNS rapidly produces amyotrophic lateral sclerosis relevant sequelae when TDP-43 is overexpressed

Year Published: 2010

Observation: transduced TDP43 with 78% transduction efficacy in rats using AAV9. Observed pronounced morbitiy, mortality and motor phenotype

Comment: TDP43 transduction is highly toxic to rats

Citation: Wang, D. B., Dayton, R. D., Henning, P. P., Cain, C. D., Zhao, L. R., Schrott, L. M., … Klein, R. L. (2010). Expansive gene transfer in the rat CNS rapidly produces amyotrophic lateral sclerosis relevant sequelae when TDP-43 is overexpressed. Molecular Therapy : The Journal of the American Society of Gene Therapy, 18(12), 2064–74. doi:10.1038/mt.2010.191

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